A California ophthalmologist and retinal surgeon is working on a drug that could potentially prevent and even reverse damage from intermediate macular degeneration. For that moonshot, she’s looking to space.
A new crew aboard the International Space Station is currently waiting for 40 very special space mice. They will be part of a payload with several other experiments to be tested aboard the ISS. The launch is scheduled for 4:55 p.m. ET Thursday, and planned docking with the ISS would be Saturday.
Dr. Hema Ramkumar cares for patients at Providence St. Jude Medical Center in Fullerton, California. She has been researching macular degeneration for 17 years.
With the disease, deposits collect and cells in the center of the eye, called the macula, break down. The retina, which sends electrical signals to the brain about what is seen, doesn’t work right. Patients can have sudden blurry vision, trouble seeing clearly at night, and eventually become blind. Ramkumar is developing a gene therapy to teach certain eye cells to self-heal.
“There is a huge impact on quality of life. For patients suffering from macular degeneration in the intermediate stage and advanced stages, this takes away people’s ability to read their own medications, do their own finances and their independence,” she told Scripps News. “For the intermediate stage, which is what we’re targeting, the patients that have full independence and are looking at their future and don’t want it to get worse. That’s what our therapy is geared at.”
As part of her research and inspired by her aerospace engineer father, Ramkumar will work with scientists aboard the International Space Station who will care for the 40 mice.
Another 40 stay on land as ground control for a month. Animals are used in research for understanding disease, and microgravity is key.
“You’ve seen their hair, [floating] in interviews. That is also happening to the fluids inside their bodies. This affects the eyes and the pressure within the eyes. And it can cause damage that mimics macular degeneration and other diseases on Earth, ” Kristin Kopperud, science program director, biological sciences at the International Space Station National Laboratory told Scripps News.
In space, spaceflight-associated neuro-ocular syndrome (SANS) can happen to astronauts after six months.
“Takes a lot longer to get to Mars. So someday we’ll be on a rocket for much longer,” said Kopperud.
Retinal scans of the mice will help inform clinical trials and one day help patients.
“I think we have a good chance. And if not, it was worth trying. And we’ll learn more, iterate. And patients need better options. That is my driving factor, ” said Ramkumar, who has founded a company, Oculogenex, for the work.
Other researchers are also working on gene therapies for advanced macular degeneration. This would be the first for patients less severe, if it works. After the mice, Ramkumar hopes for human trials sometime next year.
